The US FDA is investigating the need for further regulatory action following the deaths of two patients treated with Roche/Sarepta’s gene therapy, Elevidys, for Duchenne muscular dystrophy.
Marketing authorization applications that are underway elsewhere are geared towards ambulatory patients, not non-ambulatory patients, the group in which the deaths occurred.
Roche’s global filing strategy for the treatment was influenced by factors including whether different authorities used FDA approvals for decision making.

How Will Regulatory Filing Winds Blow Globally For Elevidys Following Patient Deaths?

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